NCT05693909 A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic Syndromes
| NCT ID | NCT05693909 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Pharmacosmos A/S |
| Condition | Beta Thalassemia Major Anemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 90 participants |
| Start Date | 2023-09-04 |
| Primary Completion | 2027-10 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 90 participants in total. It began in 2023-09-04 with a primary completion date of 2027-10.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
The goal of this clinical trial is to learn about SP-420 ability to remove iron from organs in subjects with transfusion-dependent β-thalassemia or transfusion-dependent low-risk myelodysplastic syndrome. The main questions it aims to answer are: * How efficient is SP-420 in cleaning iron from the liver? * How is the safety and tolerability of ascending doses of SP-420? Participants will: * Take medication three times weekly * Attend up to 20 site visits * Undergo MRI scans
Eligibility Criteria
Thalassemia cohorts: Inclusion criteria: * Women and men aged 18 years or older * Transfusion-dependent β-thalassemia including HbE/β-thalassemia requiring iron chelation therapy (β-thalassemia with mutation and/or multiplication of α-globin is allowed) * On a stable dose of iron chelation for at least 4 weeks prior to screening * Weight ≥ 35kg at screening * Transfusion iron overload * Treated and followed for at least the past 6 months in a specialized centre Exclusion criteria: * β-thalassemia with the structural Hb variants HbS and HbC * Current MDS * Current biliary disorder * Historic or ongoing clinically significant kidney disease * Unable to undergo trial assessments including MRI e.g. due to claustrophobia in MRI scanner * Pregnant or nursing women * Men who do not agree to practice effective barrier contraception during the entire period Myelodysplastic Syndromes Cohorts: Inclusion criteria: * Women and men aged 18 years or older * Very low, low, or intermediate risk Myelodysplastic Syndrome according to IPSS-R * Weight ≥ 35kg at screening * Transfusion iron overload * Treated and followed for at least the past 6 months at medical facilities experienced with MDS Exclusion criteria: * Therapy-related MDS or MDS with a known bone marrow fibrosis * Diagnosis of decompensated liver cirrhosis * Clinically significant kidney disease, either historic or ongoing * Uncontrolled ischemic heart disease or uncontrolled arrythmia * Uncontrolled hypertension * Uncontrolled dyslipidaemia * Uncontrolled Diabetes * Major surgery within 8 weeks prior to screening * Pregnant or nursing women * Men who do not agree to practice effective barrier contraception during the entire period
Contact & Investigator
Pharmacosmos Clinical and non-clinical Department
STUDY DIRECTOR
Pharmacosmos A/S
Frequently Asked Questions
Who can join the NCT05693909 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Beta Thalassemia Major Anemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT05693909 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT05693909 currently recruiting?
Yes, NCT05693909 is actively recruiting participants. Contact the research team at info@pharmacosmos.com for enrollment information.
Where is the NCT05693909 trial being conducted?
This trial is being conducted at Copenhagen, Denmark.
Who is sponsoring the NCT05693909 clinical trial?
NCT05693909 is sponsored by Pharmacosmos A/S. The principal investigator is Pharmacosmos Clinical and non-clinical Department at Pharmacosmos A/S. The trial plans to enroll 90 participants.